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denufosol tetrasodium

Phase 2

Cystic Fibrosis | Small molecule | Respiratory |Merck & Company, Inc.|Last Updated: Oct 20, 2015

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
RandomizedDouble-BlindPLACEBO_CONTROLLEDDMCBiomarker
Total Trials4
Total Enrollment200
FDA Designations
No designations recorded
Clinical Trials (4)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT01181622A Safety and Tolerability Study of Denufosol in 2-4 Year OldsPHASE2 COMPLETED 25 — — Aug 1, 2010Oct 1, 2010Jan 13, 2015 - —
NCT00130182A Study in Pediatric Patients With Cystic Fibrosis Lung DiseasePHASE2 COMPLETED 13 — — Aug 1, 2005 -May 22, 20133 United States
NCT00103714Study of Denufosol Tetrasodium in Patients With Cystic Fibrosis Lung DiseasePHASE2 COMPLETED 72 — — Jan 1, 2005Aug 1, 2005Oct 20, 2015 - —
NCT00056147Study of INS37217 Inhalation Solution in Mild to Moderate Cystic Fibrosis Lung DiseasePHASE2 COMPLETED 90 — — Apr 1, 2003Feb 1, 2004Jan 29, 2015 - —
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Study Endpoints
Primary Endpoints
Intolerability to study drug as measured by: oxyhemoglobin saturation, treatment-emergent cough, and new wheezes or crackles
Day 1, Day 7
respiratory function
lung function
respiratory symptoms
sputum weight
pulmonary exacerbations
measures of lung characteristics
Secondary Endpoints
Mean change from pre-dose in oxyhemoglobin saturation at defined times post-dose
Day 1, Day 7
Mean change from baseline in oxyhemoglobin saturation
Day 7
Incidence of treatment-emergent adverse events (TEAEs), serious adverse events (SAEs) and/or withdrawals due to TEAEs
Day 7
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Study Design & Arms
AllocationRANDOMIZED
MaskingDOUBLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
denufosol tetrasodium Inhalation SolutionEXPERIMENTAL -
PlaceboPLACEBO_COMPARATOR -
Interventions
NameTypeDescription
denufosol tetrasodium Inhalation SolutionDRUG60 mg by oral inhalation three times daily
0.9% w/v sodium chloride solutionDRUG0.9% w/v sodium chloride solution by oral inhalation three times daily
denufosol tetrasodium (INS37217)DRUG -
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Eligibility Criteria
Age Range2 Years — 4 Years
SexALL
Healthy VolunteersNo

Inclusion Criteria: * Have a confirmed diagnosis of CF * Have oxyhemoglobin saturation ≥ 95% prior to randomization Exclusion Criteria: * Have acute intercurrent respiratory infection (cough, wheezing, or new rhinorrhea or nasal congestion) * Have any significant medical condition not related t...

Countries:United States
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Competitive Landscape -Cystic Fibrosis 18 trials
CompanyTickerTrialsLead PhaseDrugs
Vertex Pharmaceuticals IncorporatedVRTX9PHASE3VX-121/TEZ/D-IVA, ELX/TEZ/IVA, IVA, VNZ/TEZ/D-IVA, VX-522 mRNA therapy
Sionna Therapeutics, Inc.SION2PHASE2SION-719, SION-451, SION-2222, SION-109
BiomX LtdPHGE1PHASE2BX004
4D Molecular Therapeutics, Inc.FDMT1PHASE24D-710
Arcturus Therapeutics Holdings, Inc.ARCT1PHASE2ARCT-032
Krystal Biotech, Inc.KRYS1PHASE1KB407
Illumina, Inc.ILMN1 — Undisclosed
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